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A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

NCT04257929 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 65

Last updated 2026-05-12

Study results available
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Summary

The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.

Conditions

Interventions

DRUG

Pitolisant oral tablets

Pitolisant 4.45 mg or 17.8 mg tablets

DRUG

Placebo oral tablet

Matching placebo tablets

Sponsors & Collaborators

  • Harmony Biosciences Management, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-09
Primary Completion
2022-08-17
Completion
2026-01-21
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04257929 on ClinicalTrials.gov